PEP-CTN EHR to RAVE supplement

PEP-CTN EHR 至 RAVE 补充

• 批准号: 10440223
• 负责人: Brenda J. Weigel
• 金额: $ 63.85万
• 依托单位: PUBLIC HEALTH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-09-20 至 2023-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10440223
• 关键词: Adolescent; Australia; Biological; Biology; Canada; Characteristics; Child; Childhood; Clinical Research; Clinical Trials; Clinical Trials Network; Cohort Studies; Common Terminology Criteria for Adverse Events; Complex; Data; Data Collection; Data Security; Data Set; Data Storage and Retrieval; Databases; Development; Drug Kinetics; Enrollment; Ensure; Environment; Future; Genes; Genomics; Individual; Informatics; Infrastructure; Institution; International; Laboratories; Laboratory Research; Laboratory Study; Malignant Childhood Neoplasm; Malignant Neoplasms; Maps; Medidata; Mission; Modeling; New Agents; Parents; Patients; Pediatric Oncology; Pediatric Oncology Group; Peer Review; Pharmacogenetics; Pharmacology; Pharmacology Study; Phase; Pilot Projects; Process; Protocols documentation; Reporting; Research; Resources; Role; Signal Transduction; Site; System; Testing; Therapeutic; TimeLine; Update; cancer therapy; data exchange; early phase clinical trial; exome sequencing; imaging modality; imaging study; member; novel anticancer drug; operation; pilot trial; response; skills; systems research; targeted agent; targeted treatment; tool; transcriptome sequencing; tumor; tumor DNA

PEP-CTN Project Summary/Abstract: The mission of the Pediatric Early Phase Clinical Trials Network (PEP-CTN) is to identify and develop effective new agents for children and adolescents with cancer, through rational and efficient clinical and laboratory research. PEP-CTN clinical trials will incorporate correlative genomics, biology, pharmacology, and imaging studies to further the understanding of the disposition and action of new agents introduced into the treatment of children with cancer. Annually, the PEP-CTN will enroll approximately 120 children and adolescents with cancer onto clinical trials of novel anticancer agents at the 21 core-member sites and 21 non-core member sites throughout the US, Canada, and Australia. The network institutions are selected through a peer review process, and serve as a national and international model for new agent development in pediatric oncology. The PEP-CTN leverages the database infrastructure and resources of the parent Children’s Oncology Group while maintaining its own administrative and operational infrastructure to ensure rapid development, implementation, and reporting of specialized and complex early phase clinical trials. The PEP-CTN has expertise and resources for the conduct of translational biology, pharmacokinetic, and pharmacogenetic studies, and utilizes state-of-the-art informatics systems to facilitate the transfer of response and correlative imaging studies for central review and analyses. The PEP-CTN's primary specific aims are: 1) To safely and efficiently introduce novel anticancer agents into the pediatric setting through the conduct of early phase clinical trials; 2) To expeditiously obtain preliminary efficacy signals through use of phase 2 expansion cohorts and pilot studies in order to inform tumor specific trials that will be conducted across COG sites; 3) To perform genomic analyses, including single gene studies or gene panels, to identify appropriate patients for early phase studies of targeted agents; 4) To identify associations of tumor characteristics with response to new agents using genomic analyses such as whole exome sequencing and RNA sequencing; 5) To incorporate pharmacologic and biologic endpoints, including circulating tumor DNA, other translational laboratory studies, and imaging modalities, into early phase studies, in order to enhance our understanding of the new agents and their effect on tumors.. Development of targeted therapy for childhood cancer is a high priority as it offers the prospect of more efficacious and less toxic therapeutics.

PEP-CTN 项目摘要/摘要： 儿科早期临床试验网络 (PEP-CTN) 的使命是识别和 通过合理和有效的方法，开发治疗儿童和青少年癌症的有效新药 高效的临床和实验室研究。 PEP-CTN 临床试验将纳入相关的 基因组学、生物学、药理学和影像学研究，以进一步了解 引入治疗癌症儿童的新药物的处置和作用。 每年，PEP-CTN 都会招收大约 120 名患有癌症的儿童和青少年。 新型抗癌药物在21个核心成员和21个非核心成员的临床试验 站点遍布美国、加拿大和澳大利亚。网络机构是通过 同行评审过程，并作为新制剂开发的国家和国际典范 在儿科肿瘤学中。 PEP-CTN 利用数据库基础设施和资源 母公司儿童肿瘤学小组，同时维持其自己的行政和运营 确保快速开发、实施和报告专业和 复杂的早期临床试验。 PEP-CTN 拥有开展以下活动的专业知识和资源： 转化生物学、药代动力学和药物遗传学研究，并利用最先进的技术 信息学系统，以促进反应和相关成像研究的转移 中央审查和分析。 PEP-CTN 的主要具体目标是： 1) 安全有效地引入新型抗癌药物 通过进行早期临床试验将药物引入儿科环境； 2) 至 通过使用 2 期扩展队列快速获得初步疗效信号， 试点研究，以便为将在 COG 站点进行的肿瘤特异性试验提供信息； 3) 至 进行基因组分析，包括单基因研究或基因组，以确定合适的 接受靶向药物早期研究的患者； 4) 识别肿瘤的关联 使用基因组分析（例如全外显子组）对新药物的反应特征 测序和RNA测序； 5) 结合药理学和生物学终点， 包括循环肿瘤 DNA、其他转化实验室研究和成像方式， 进入早期研究，以增强我们对新药及其效果的了解 儿童癌症靶向治疗的开发是当务之急，因为它提供了 更有效、毒性更小的治疗方法的前景。